Ny studie på Spinraza® (nusinersen) för behandling av spädbarn med Spinal Muskel Atrofi

Ny studie på Spinraza® (nusinersen) för behandling av spädbarn med Spinal Muskel Atrofi

NURTURE-studien publiceras i tidskriften Neuromuscular Disorders. I studien undersöktes presymptomatisk behandling med Spinraza® (nusinersen) av spädbarn med Spinal Muskel Atrofi

 

  • Behandling med Spinraza innebar en 100 procentig överlevnad utan att någon patient behövde permanent ventilationshjälp.
  • Milstolpar för motorisk funktion som uppnåddes var att 100 procent av studiedeltagarna kunde sitta utan stöd och att 88 procent klarade att gå på egen hand. Dessa resultat skiljer sig avsevärt mot vad man kan förvänta sig utifrån sjukdomens naturliga förlopp.
  • Studieresultaten bekräftar den befintliga säkerhetsprofilen och den dokumenterade effekten av Spinraza som är den enda godkända behandlingen av SMA hos spädbarn, barn och vuxna.

 

Biogen today announced that the journal Neuromuscular Disorders has published data from NURTURE, the first study investigating a treatment targeting the underlying cause of spinal muscular atrophy (SMA) in infants treated pre-symptomatically. Data from the NURTURE study demonstrated that infants who initiated treatment with nusinersen prior to the onset of clinical symptoms attained unparalleled results compared to the natural history of the disease. As of March 2019 all participants were alive, without the need for permanent ventilation and experienced continuous improvements with the majority achieving motor milestones in timelines consistent with normal development. The results also demonstrated durability of effect with children making progress throughout the study.

NURTURE is an ongoing, Phase 2, open-label study of 25 infants with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of nusinersen in the pre-symptomatic stage and before six weeks old. The study, conducted at 15 sites in seven countries, has results up to 45.4 months. When compared with the natural history of the disease, the results are significant in their impact on changing the course of SMA.

As of March 2019 all infants in the study were 25 months or older, past the typical age of symptom onset for SMA Type 1 and Type 2, and were alive without the need for permanent ventilation. In comparison to the natural history of SMA, many of these infants would likely have passed away or require permanent ventilation on average by 13.5 months. In both the children with two and three copies of SMN2, treatment with Spinraza demonstrated rapid onset of improvement and durability of effect with their mean Children’s Hospital of Pennsylvania Infant Test of Neuromuscular Disorders (CHOP-INTEND) score of motor function reaching the maximum mean score of 64 for all participants with three copies of SMN2 (n=10) and a mean of 62.1 for those with two copies of SMN2 (N=15).

Additional highlights include:

  • The majority of study participants achieved motor milestones in timeframes consistent with the World Health Organization (WHO) standards, with 100 percent sitting independently and 88 percent walking independently.
  • Hammersmith Infant Neurologic Examination, Section 2 (HINE-2) development of motor function scores increased for all participants with the mean score for both those with two or three SMN2 copies approaching the maximum score of 26 points at the last assessment.
  • SPINRAZA demonstrated longer term efficacy up to nearly four years, with participants continuing to make progress.
  • SPINRAZA was well-tolerated with no new safety concerns identified after up to nearly four years of treatment.

These published results from the NURTURE study were previously presented at the 2019 Cure SMA Annual SMA Conference and the 5th Congress of the European Academy of Neurology.

Om Spinraza
Spinraza är avsett för behandling av spinal muskelatrofi av typ 5q. Biverkningar som observerats är fall av allvarlig infektion, såsom meningit. Det har även förekommit rapporter om kommunicerande hydrocefalus, aseptisk meningit och överkänslighet (t.ex. angioödem, urtikaria och hudutslag). För information om kontraindikationer, varningar och försiktighet, biverkningar, dosering och förpackningar se www.fass.se.

References

1 Darras BT, Chiriboga CA, Iannaccone ST, et al. Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies. Neurology. 2019 May 21;92(21):e2492-e2506.

2 Finkel R, Chiriboga C, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016;388(10063):3017-3026.

3 Darras B, Jones Jr. H.R., Ryan M., De Vivo D. Chapter 8 – Spinal Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of Infancy, Childhood, and Adolescence (Second Edition). San Diego: Academic Press; 2015:117-145.